Fredwall, Svein http://orcid.org/0000-0002-2804-5783
AlSayed, Moeenaldeen
Ben-Omran, Tawfeg
Boero, Silvio
Cormier-Daire, Valérie
Fauroux, Brigitte
Guillén-Navarro, Encarna
Innig, Florian
Kunkel, Philip
Lampe, Christian
Maghnie, Mohamad http://orcid.org/0000-0002-7183-5238
Mohnike, Klaus
Mortier, Geert http://orcid.org/0000-0001-9871-4578
Pejin, Zagorka
Sessa, Marco
Sousa, Sérgio B. http://orcid.org/0000-0003-3921-1334
Irving, Melita
Funding for this research was provided by:
BioMarin Pharmaceutical
Sanofi
Ascendis
QED Therapeutics
Article History
Received: 22 March 2024
Accepted: 19 April 2024
First Online: 15 May 2024
Declarations
:
: Svein Fredwall, Moeenaldeen AlSayed, Tawfeg Ben Omran, Brigitte Fauroux and Christian Lampe have nothing to disclose. Silvio Boero has received payment or honoraria, support for attending meetings and/or travel from BioMarin. Valerie Cormier-Daire has received payment or honoraria, support for attending meetings and/or travel from BioMarin. Sérgio B Sousa has received payment or honoraria, support for attending meetings and/or travel and has participated in Advisory Boards for BioMarin. Encarna Guillen-Navarro has received payment or honoraria, support for attending meetings and/or travel from BioMarin. Florian Innig has received grants or contracts paid to BKMF e.V from BioMarin. Melita Irving has received grants or contracts and consulting fees from BioMarin, QED/Bridge Bio, Ascendis, Sanofi, Tyra, NovoNordisk; and payment or honoraria from BioMarin; and has participated in Advisory Boards for BioMarin and QED/BridgeBio. Philip Kunkel has received payment or honoraria, support for attending meetings and/or travel from BioMarin. Mohamad Maghnie has received consulting fees, payment or honoraria, support for attending meetings and/or travel and has participated in Advisory Boards for BioMarin. Klaus Mohnike has received consulting fees from BioMarin and NovoNordisk, payment or honoraria from Kyowa-Kirin, support for attending meetings and/or travel from BioMarin, has participated in Advisory Boards for QED and is Vice-Chair of BKMJ. Geert Mortier has received payment or honoraria and support for attending meetings and/or travel from BioMarin, and has participated in Advisory Boards for Sanofi. Zagorka Pejin has received consulting fees and payment or honoraria from Orthofix Italy and Newclip France; support for attending meetings and/or travel from Orthofix Italy, and patents planned, issued or pending with Orthofix Italy. Marco Sessa has received grants or contracts paid to AISAC OdV from BioMarin and QED.
: This article is based on previously conducted studies and does not contain any new studies with human participants or animals performed by any of the authors.